Trial Outcomes & Findings for Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT NCT03500549)

NCT ID: NCT03500549

Last Updated: 2022-03-25

Results Overview

Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 80 participants
• Primary outcome timeframe: Baseline and Week 16
• Results posted on: 2022-03-25

Participant Flow

This Phase 3, randomized, multicenter, open-label, active-comparator controlled study evaluated the efficacy and safety of pegcetacoplan (APL-2) in subjects with paroxysmal nocturnal hemoglobinuria who had received treatment with eculizumab (Soliris®) in the past.

The treatment period of the study consisted of 3 parts: a 4-week run-in period, a 16-week randomized controlled period (RCP), and a 32-week open-label period. Of the 102 subjects screened, 80 subjects met all the inclusion criteria and none of the exclusion criteria and entered the run-in period. Randomization was stratified by the number of packed red blood cell (PRBC) transfusions within the 12 months prior to Day -28 and platelet count at screening.

Participant milestones

Measure	Pegcetacoplan to Pegcetacoplan During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly subcutaneous (SC) doses of pegcetacoplan 1080 milligrams (mg) in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).
Run-in Period (Day -28 to ≤Day 1) STARTED	41	39
Run-in Period (Day -28 to ≤Day 1) COMPLETED	41	39
Run-in Period (Day -28 to ≤Day 1) NOT COMPLETED	0	0
RCP (Day 1 - Week 16) STARTED	41	39
RCP (Day 1 - Week 16) COMPLETED	38	39
RCP (Day 1 - Week 16) NOT COMPLETED	3	0
Open-label Period (Week 17 to Week 48) STARTED	38	39
Open-label Period (Week 17 to Week 48) COMPLETED	35	32
Open-label Period (Week 17 to Week 48) NOT COMPLETED	3	7

Reasons for withdrawal

Measure	Pegcetacoplan to Pegcetacoplan During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly subcutaneous (SC) doses of pegcetacoplan 1080 milligrams (mg) in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).
RCP (Day 1 - Week 16) Adverse Event	3	0
Open-label Period (Week 17 to Week 48) Adverse Event	3	7

Baseline Characteristics

Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Baseline characteristics by cohort

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Total n=80 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	31 Participants n=5 Participants	32 Participants n=7 Participants	63 Participants n=5 Participants
Age, Categorical >=65 years	10 Participants n=5 Participants	7 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Female	27 Participants n=5 Participants	22 Participants n=7 Participants	49 Participants n=5 Participants
Sex: Female, Male Male	14 Participants n=5 Participants	17 Participants n=7 Participants	31 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	29 Participants n=5 Participants	32 Participants n=7 Participants	61 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	10 Participants n=5 Participants	6 Participants n=7 Participants	16 Participants n=5 Participants
Race/Ethnicity, Customized Asian	5 Participants n=5 Participants	7 Participants n=7 Participants	12 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized White	24 Participants n=5 Participants	25 Participants n=7 Participants	49 Participants n=5 Participants
Race/Ethnicity, Customized Other	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Not Reported	10 Participants n=5 Participants	6 Participants n=7 Participants	16 Participants n=5 Participants
Number of transfusions in the last 12 months prior to Day -28 < 4	20 Participants n=5 Participants	16 Participants n=7 Participants	36 Participants n=5 Participants
Number of transfusions in the last 12 months prior to Day -28 ≥ 4	21 Participants n=5 Participants	23 Participants n=7 Participants	44 Participants n=5 Participants
Platelet count at screening <100,000 (count/mm^3)	12 Participants n=5 Participants	9 Participants n=7 Participants	21 Participants n=5 Participants
Platelet count at screening ≥100,000 (count/mm^3)	29 Participants n=5 Participants	30 Participants n=7 Participants	59 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP	2.37 Grams per deciliter (g/dL) Standard Error 0.363	-1.47 Grams per deciliter (g/dL) Standard Error 0.666	—

SECONDARY outcome

Timeframe: Day 1 to Week 16

Population: The ITT set included all randomized subjects.

Subjects who experienced more than 1 transfusion during the RCP are only counted once. Subjects who did not have a transfusion but withdrew before Week 16 were considered as having a transfusion in the analysis of transfusion avoidance.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Percentage of Subjects Who Did Not Require a Transfusion (Transfusion Avoidance) During the RCP	85.4 Percentage of subjects	15.4 Percentage of subjects	—

SECONDARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
LS Mean Change From Baseline to Week 16 in Absolute Reticulocyte Count (ARC) During the RCP	-135.82 10^9 cells/ liter (L) Standard Error 6.543	27.79 10^9 cells/ liter (L) Standard Error 11.859	—

SECONDARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
LS Mean Change From Baseline to Week 16 in Lactate Dehydrogenase (LDH) Level During the RCP	-14.76 Units (U)/L Standard Error 42.708	-10.12 Units (U)/L Standard Error 71.025	—

SECONDARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

The FACIT-fatigue scale version 4 is a 13-item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher quality of life (QoL). Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
LS Mean Change From Baseline to Week 16 in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Score During the RCP	9.22 Score on a scale Standard Error 1.607	-2.65 Score on a scale Standard Error 2.821	—

SECONDARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

Hb response was defined as an increase of at least 1 g/dL in Hb from Baseline at Week 16. Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Percentage of Subjects Who Achieved a Hb Response in the Absence of Transfusions at Week 16	75.6 Percentage of subjects	0.0 Percentage of subjects	—

SECONDARY outcome

Timeframe: Week 16

Population: The ITT set includes all randomized subjects.

Reticulocyte normalization was defined as the ARC being below the upper limit of the gender-specific normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 were classified as nonresponders.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Percentage of Subjects Who Achieved Reticulocyte Normalization in the Absence of Transfusions at Week 16	78.0 Percentage of subjects	2.6 Percentage of subjects	—

SECONDARY outcome

Timeframe: Week 16

Population: The ITT set included all randomized subjects.

Hb normalization was defined as the Hb level being above the lower limit of the normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 are classified as nonnormalization.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Percentage of Subjects Who Achieved Hb Normalization in the Absence of Transfusions at Week 16	34.1 Percentage of subjects	0.0 Percentage of subjects	—

SECONDARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
LS Mean Change From Baseline to Week 16 in Indirect Bilirubin Level During the RCP	-17.78 Micromole (μmol)/L Standard Error 2.727	4.15 Micromole (μmol)/L Standard Error 4.477	—

SECONDARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
LS Mean Change From Baseline to Week 16 in Haptoglobin Level During the RCP	-0.02 g/L Standard Error 0.033	0.12 g/L Standard Error 0.063	—

SECONDARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
LS Mean Change From Baseline to Week 16 in Linear Analog Scale Assessment (LASA) Scores During the RCP	49.38 Score on a scale Standard Error 10.189	-9.72 Score on a scale Standard Error 18.988	—

SECONDARY outcome

Timeframe: Baseline and Week 16

Population: The ITT set included all randomized subjects.

The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are "Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Global Health Status/QoL	15.91 Score on a scale Standard Error 3.635	-2.71 Score on a scale Standard Error 8.515	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Functional Scales - Physical functioning	16.92 Score on a scale Standard Error 2.081	4.06 Score on a scale Standard Error 3.605	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Functional Scales - Role functioning	15.39 Score on a scale Standard Error 3.930	-9.04 Score on a scale Standard Error 6.954	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Functional Scales - Emotional functioning	7.98 Score on a scale Standard Error 3.366	3.86 Score on a scale Standard Error 7.237	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Functional Scales - Cognitive functioning	5.76 Score on a scale Standard Error 3.258	-3.80 Score on a scale Standard Error 6.420	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Functional Scales - Social functioning	15.08 Score on a scale Standard Error 2.946	3.82 Score on a scale Standard Error 6.349	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Fatigue	-22.93 Score on a scale Standard Error 3.321	-2.18 Score on a scale Standard Error 6.644	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Nausea and vomiting	-0.34 Score on a scale Standard Error 1.632	-0.33 Score on a scale Standard Error 3.876	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Pain	-0.74 Score on a scale Standard Error 4.323	2.01 Score on a scale Standard Error 7.841	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Dyspnoea	-20.12 Score on a scale Standard Error 3.488	-5.55 Score on a scale Standard Error 7.019	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Insomnia	-9.18 Score on a scale Standard Error 3.955	-9.50 Score on a scale Standard Error 7.090	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Appetite loss	-3.76 Score on a scale Standard Error 3.357	4.19 Score on a scale Standard Error 7.009	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Constipation	2.98 Score on a scale Standard Error 3.248	1.19 Score on a scale Standard Error 8.129	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Diarrhoea	0.31 Score on a scale Standard Error 3.711	1.68 Score on a scale Standard Error 8.204	—
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP Symptom Scales - Financial difficulties	-6.82 Score on a scale Standard Error 3.853	0.58 Score on a scale Standard Error 6.297	—

SECONDARY outcome

Timeframe: Day 1 to Week 16

Population: The ITT set included all randomized subjects.

Subjects who withdrew during the RCP before Week 16 will have their number of units of PRBC estimated from the duration they were in the study.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=41 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Total Number of PRBC Units Transfused During the RCP	26 PRBC units	198 PRBC units	—

SECONDARY outcome

Timeframe: Baseline and Week 48

Population: The ITT set included all randomized subjects.

Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=33 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=30 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Baseline to Week 48 in Hb Level During the Treatment Period	2.47 g/dL Standard Deviation 1.72	2.93 g/dL Standard Deviation 2.09	—

SECONDARY outcome

Timeframe: Week 17 and Week 48

Population: The ITT set included all randomized subjects.

Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=33 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=29 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Week 17 to Week 48 in Hb Level During the Open-label Period	-0.16 g/dL Standard Deviation 1.154	2.89 g/dL Standard Deviation 2.078	—

SECONDARY outcome

Timeframe: Baseline and Week 48

Population: The ITT set included all randomized subjects.

Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=31 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=29 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Baseline to Week 48 in ARC During the Treatment Period	-135.64 10^9 cells/L Standard Deviation 67.90	-128.22 10^9 cells/L Standard Deviation 59.60	—

SECONDARY outcome

Timeframe: Week 17 and Week 48

Population: The ITT set included all randomized subjects.

Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=31 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=29 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Week 17 to Week 48 in ARC During the Open-label Period	-6.50 10^9 cells/L Standard Deviation 26.471	-121.15 10^9 cells/L Standard Deviation 70.969	—

SECONDARY outcome

Timeframe: Baseline and Week 48

Population: The ITT set included all randomized subjects.

Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=33 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=30 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Baseline to Week 48 in LDH Level During the Treatment Period	-41.53 U/L Standard Deviation 153.68	-105.27 U/L Standard Deviation 315.59	—

SECONDARY outcome

Timeframe: Week 17 and Week 48

Population: The ITT set included all randomized subjects.

Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=33 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=28 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Week 17 to Week 48 in LDH Level During the Open-label Period	8.03 U/L Standard Deviation 129.285	-46.84 U/L Standard Deviation 292.607	—

SECONDARY outcome

Timeframe: Baseline and Week 48

Population: The ITT set included all randomized subjects.

The FACIT-fatigue scale version 4 is a 13-item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher QoL. Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=30 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=29 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Baseline to Week 48 in FACIT-Fatigue Scale Score During the Treatment Period	10.14 Score on a scale Standard Deviation 9.06	9.62 Score on a scale Standard Deviation 10.34	—

SECONDARY outcome

Timeframe: Week 17 and Week 48

Population: The ITT set included all randomized subjects.

The FACIT-fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher QoL.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=30 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=26 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Week 17 to Week 48 in FACIT-Fatigue Scale Score During the Open-label Period	1.28 Score on a scale Standard Deviation 7.805	10.19 Score on a scale Standard Deviation 10.973	—

SECONDARY outcome

Timeframe: Baseline and Week 48

Population: The ITT set included all randomized subjects.

The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=29 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=29 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Baseline to Week 48 in LASA Scores During the Treatment Period	58.66 Score on a scale Standard Deviation 51.16	56.52 Score on a scale Standard Deviation 65.55	—

SECONDARY outcome

Timeframe: Week 17 and Week 48

Population: The ITT set included all randomized subjects.

The FACIT-fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher QoL.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=30 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=26 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Week 17 to Week 48 in LASA Scores During the Open-label Period	13.13 Score on a scale Standard Deviation 46.296	62.92 Score on a scale Standard Deviation 60.053	—

SECONDARY outcome

Timeframe: Baseline and Week 48

Population: The ITT set included all randomized subjects.

The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are 'Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=30 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=28 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Global Health Status/QoL	18.89 Score on a scale Standard Deviation 17.635	13.99 Score on a scale Standard Deviation 22.912	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Functional Scales - Physical functioning	15.33 Score on a scale Standard Deviation 15.278	10.80 Score on a scale Standard Deviation 17.765	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Functional Scales - Role functioning	16.67 Score on a scale Standard Deviation 27.334	20.11 Score on a scale Standard Deviation 27.595	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Functional Scales - Emotional functioning	10.28 Score on a scale Standard Deviation 18.657	5.36 Score on a scale Standard Deviation 17.005	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Functional Scales - Cognitive functioning	7.78 Score on a scale Standard Deviation 23.462	0.00 Score on a scale Standard Deviation 18.703	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Functional Scales - Social functioning	16.11 Score on a scale Standard Deviation 24.166	14.88 Score on a scale Standard Deviation 22.379	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Fatigue	-21.48 Score on a scale Standard Deviation 26.733	-23.75 Score on a scale Standard Deviation 29.506	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Nausea and vomiting	-2.22 Score on a scale Standard Deviation 11.357	0.00 Score on a scale Standard Deviation 4.454	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Pain	0.56 Score on a scale Standard Deviation 27.849	3.45 Score on a scale Standard Deviation 20.596	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Dyspnoea	-17.78 Score on a scale Standard Deviation 29.985	-27.59 Score on a scale Standard Deviation 33.415	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Insomnia	-6.67 Score on a scale Standard Deviation 25.371	0.00 Score on a scale Standard Deviation 28.172	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Appetite loss	-7.78 Score on a scale Standard Deviation 14.339	-3.45 Score on a scale Standard Deviation 22.440	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Constipation	-1.11 Score on a scale Standard Deviation 22.289	-2.38 Score on a scale Standard Deviation 8.742	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Diarrhoea	1.11 Score on a scale Standard Deviation 29.664	5.95 Score on a scale Standard Deviation 15.853	—
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period Symptom Scales - Financial difficulties	-15.56 Score on a scale Standard Deviation 24.343	-8.33 Score on a scale Standard Deviation 19.510	—

SECONDARY outcome

Timeframe: Week 17 and Week 48

Population: The ITT set included all randomized subjects.

The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are 'Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=30 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=26 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Global Health Status/QoL	7.22 Score on a scale Standard Deviation 19.664	23.08 Score on a scale Standard Deviation 22.149	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Functional Scales - Physical functioning	0.89 Score on a scale Standard Deviation 10.168	11.03 Score on a scale Standard Deviation 17.173	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Functional Scales - Role functioning	5.00 Score on a scale Standard Deviation 20.599	19.87 Score on a scale Standard Deviation 22.617	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Functional Scales - Emotional functioning	-2.22 Score on a scale Standard Deviation 27.328	1.92 Score on a scale Standard Deviation 13.806	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Functional Scales - Cognitive functioning	-2.78 Score on a scale Standard Deviation 16.999	2.56 Score on a scale Standard Deviation 24.355	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Functional Scales - Social functioning	3.89 Score on a scale Standard Deviation 18.919	12.18 Score on a scale Standard Deviation 23.361	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Fatigue	-2.96 Score on a scale Standard Deviation 20.824	-23.08 Score on a scale Standard Deviation 28.790	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Nausea and vomiting	-2.22 Score on a scale Standard Deviation 5.762	-4.49 Score on a scale Standard Deviation 12.072	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Pain	-2.78 Score on a scale Standard Deviation 23.195	-5.77 Score on a scale Standard Deviation 21.051	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Dyspnoea	3.33 Score on a scale Standard Deviation 25.295	-19.23 Score on a scale Standard Deviation 28.555	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Insomnia	8.89 Score on a scale Standard Deviation 23.050	-5.13 Score on a scale Standard Deviation 27.797	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Appetite loss	-8.89 Score on a scale Standard Deviation 26.164	-5.13 Score on a scale Standard Deviation 22.494	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Constipation	-1.11 Score on a scale Standard Deviation 20.498	-1.28 Score on a scale Standard Deviation 11.473	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Diarrhoea	-4.44 Score on a scale Standard Deviation 28.679	3.85 Score on a scale Standard Deviation 27.206	—
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period Symptom Scales - Financial difficulties	-2.22 Score on a scale Standard Deviation 12.172	-2.56 Score on a scale Standard Deviation 16.119	—

SECONDARY outcome

Timeframe: Week 17 to Week 48

Population: The ITT set included all randomized subjects.

Number of units of PRBC transfused to subjects in the open-label period are reported.

Outcome measures

Measure	Pegcetacoplan to Pegcetacoplan n=38 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Eculizumab to Pegcetacoplan n=39 Participants During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. On Day 1, the subjects were randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks up to the end of the RCP (Week 16). Subjects then entered the open-label run-in period where they received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20) before crossing over to monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days up to the end of the open-label period (Week 48).	Open-label Run-in Period: Crossover to Pegcetacoplan n=38 Participants Subjects in the open-label run-in period received pegcetacoplan 1080 mg twice-weekly in addition to eculizumab for 4 weeks (Week 17 to Week 20).
Total Number of PRBC Units Transfused During the Open-Label Period	68 PRBC Units	110 PRBC Units	14 PRBC Units

Adverse Events

Run-in Period: Pegcetacoplan + Eculizumab

Serious events: 4 serious events

Other events: 71 other events

Deaths: 0 deaths

Open-label Period: Pegcetacoplan

Serious events: 18 serious events

Other events: 71 other events

Deaths: 1 deaths

RCP: Eculizumab

Serious events: 5 serious events

Other events: 36 other events

Deaths: 0 deaths

RCP: Pegcetacoplan

Serious events: 7 serious events

Other events: 36 other events

Deaths: 0 deaths

Serious adverse events

Measure	Run-in Period: Pegcetacoplan + Eculizumab n=80 participants at risk During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. During the 4-week open-label run-in period (Week 17 to Week 20) subjects randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks during the RCP also received twice-weekly SC doses of pegcetacoplan 1080 mg.	Open-label Period: Pegcetacoplan n=77 participants at risk The subjects who were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days during the RCP continued to receive monotherapy with pegcetacoplan until the end of the open-label period (Week 17 to Week 48). Subjects randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks during the RCP received monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days after the open-label run-in period, up to the end of the open-label period (Week 20 to Week 48).	RCP: Eculizumab n=39 participants at risk Subjects randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks during the RCP.	RCP: Pegcetacoplan n=41 participants at risk Subjects randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days during the RCP.
Vascular disorders Deep vein thrombosis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Acute myeloid leukaemia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Diffuse large B-cell lymphoma	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Immune system disorders Allergy to immunoglobulin therapy	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Pyrexia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Hyperthermia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Reproductive system and breast disorders Ovarian cyst	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Cardiac disorders Atrial fibrillation	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Blood and lymphatic system disorders Haemolysis	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	6.5% 5/77 • Number of events 5 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Blood and lymphatic system disorders Cytopenia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Blood and lymphatic system disorders Haemolytic anaemia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Blood and lymphatic system disorders Anaemia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Respiratory, thoracic and mediastinal disorders Hypersensitivity pneumonitis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Nervous system disorders Facial paralysis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Abdominal pain	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Intestinal ischaemia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Oedematous pancreatitis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Small intestinal obstruction	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Hepatobiliary disorders Cholelithiasis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Hepatobiliary disorders Hepatocellular injury	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Hepatobiliary disorders Hyperbilirubinaemia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Hepatobiliary disorders Jaundice	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Renal and urinary disorders Acute kidney injury	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Musculoskeletal and connective tissue disorders Haematoma muscle	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations COVID-19	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Gastroenteritis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Bacterial infection	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Biliary sepsis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Diverticulitis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Post procedural sepsis	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Sepsis	2.5% 2/80 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Upper respiratory tract infection	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Nasopharyngitis	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks

Other adverse events

Measure	Run-in Period: Pegcetacoplan + Eculizumab n=80 participants at risk During the 4-week run-in period (Day -28 to ≤Day 1) all subjects received twice-weekly SC doses of pegcetacoplan 1080 mg in addition to their current dosage of eculizumab treatment. During the 4-week open-label run-in period (Week 17 to Week 20) subjects randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks during the RCP also received twice-weekly SC doses of pegcetacoplan 1080 mg.	Open-label Period: Pegcetacoplan n=77 participants at risk The subjects who were randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days during the RCP continued to receive monotherapy with pegcetacoplan until the end of the open-label period (Week 17 to Week 48). Subjects randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks during the RCP received monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days after the open-label run-in period, up to the end of the open-label period (Week 20 to Week 48).	RCP: Eculizumab n=39 participants at risk Subjects randomized to receive monotherapy with their pre-screening stable dose of eculizumab via intravenous infusion every 2 weeks during the RCP.	RCP: Pegcetacoplan n=41 participants at risk Subjects randomized to receive monotherapy with SC infusions of pegcetacoplan 1080 mg twice-weekly or every 3 days during the RCP.
Vascular disorders Hypertension	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.3% 3/41 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Injection site erythema	41.2% 33/80 • Number of events 75 • Day -28 to Week 54, a maximum of approximately 58 weeks	11.7% 9/77 • Number of events 127 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	17.1% 7/41 • Number of events 44 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Fatigue	6.2% 5/80 • Number of events 5 • Day -28 to Week 54, a maximum of approximately 58 weeks	10.4% 8/77 • Number of events 11 • Day -28 to Week 54, a maximum of approximately 58 weeks	15.4% 6/39 • Number of events 7 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Pyrexia	7.5% 6/80 • Number of events 6 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.8% 6/77 • Number of events 9 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Injection site pruritus	15.0% 12/80 • Number of events 14 • Day -28 to Week 54, a maximum of approximately 58 weeks	6.5% 5/77 • Number of events 7 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Asthenia	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.2% 4/77 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	12.8% 5/39 • Number of events 7 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.3% 3/41 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Injection site bruising	2.5% 2/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 7 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Injection site induration	6.2% 5/80 • Number of events 12 • Day -28 to Week 54, a maximum of approximately 58 weeks	6.5% 5/77 • Number of events 27 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.3% 3/41 • Number of events 8 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Injection site reaction	10.0% 8/80 • Number of events 26 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 8 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	9.8% 4/41 • Number of events 56 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Injection site swelling	12.5% 10/80 • Number of events 18 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	9.8% 4/41 • Number of events 6 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Injection site pain	7.5% 6/80 • Number of events 13 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.2% 4/77 • Number of events 68 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 9 • Day -28 to Week 54, a maximum of approximately 58 weeks
General disorders Vaccination site pain	3.8% 3/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Psychiatric disorders Anxiety	2.5% 2/80 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.2% 4/77 • Number of events 5 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Psychiatric disorders Insomnia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Injury, poisoning and procedural complications Vaccination complication	2.5% 2/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Injury, poisoning and procedural complications Contusion	3.8% 3/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.2% 4/77 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Cardiac disorders Palpitations	1.2% 1/80 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Blood and lymphatic system disorders Haemolysis	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	16.9% 13/77 • Number of events 14 • Day -28 to Week 54, a maximum of approximately 58 weeks	23.1% 9/39 • Number of events 14 • Day -28 to Week 54, a maximum of approximately 58 weeks	9.8% 4/41 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks
Blood and lymphatic system disorders Thrombocytopenia	2.5% 2/80 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 5 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Blood and lymphatic system disorders Anaemia	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	12.8% 5/39 • Number of events 5 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Respiratory, thoracic and mediastinal disorders Cough	2.5% 2/80 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	11.7% 9/77 • Number of events 9 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.8% 6/77 • Number of events 7 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.7% 3/39 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Respiratory, thoracic and mediastinal disorders Dyspnoea	5.0% 4/80 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.7% 3/39 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Nervous system disorders Headache	12.5% 10/80 • Number of events 12 • Day -28 to Week 54, a maximum of approximately 58 weeks	10.4% 8/77 • Number of events 13 • Day -28 to Week 54, a maximum of approximately 58 weeks	23.1% 9/39 • Number of events 10 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Nervous system disorders Dizziness	3.8% 3/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	12.8% 5/39 • Number of events 5 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Nervous system disorders Lethargy	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Diarrhoea	12.5% 10/80 • Number of events 11 • Day -28 to Week 54, a maximum of approximately 58 weeks	14.3% 11/77 • Number of events 15 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	22.0% 9/41 • Number of events 9 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Abdominal pain	2.5% 2/80 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.7% 3/39 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	9.8% 4/41 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Abdominal distension	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.2% 4/77 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Nausea	8.8% 7/80 • Number of events 8 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Vomiting	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	10.3% 4/39 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Abdominal discomfort	2.5% 2/80 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/77 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Gastrointestinal disorders Constipation	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.7% 3/39 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Renal and urinary disorders Acute kidney injury	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.2% 4/77 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Renal and urinary disorders Chromaturia	3.8% 3/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Renal and urinary disorders Haemoglobinuria	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	1.3% 1/77 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Hepatobiliary disorders Hyperbilirubinaemia	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Skin and subcutaneous tissue disorders Erythema	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Musculoskeletal and connective tissue disorders Arthralgia	2.5% 2/80 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.8% 6/77 • Number of events 7 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Musculoskeletal and connective tissue disorders Pain in extremity	3.8% 3/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	6.5% 5/77 • Number of events 6 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 5 • Day -28 to Week 54, a maximum of approximately 58 weeks
Musculoskeletal and connective tissue disorders Back pain	3.8% 3/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	10.3% 4/39 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.3% 3/41 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks
Musculoskeletal and connective tissue disorders Myalgia	3.8% 3/80 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.2% 4/77 • Number of events 4 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Metabolism and nutrition disorders Decreased appetite	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 2/77 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Nasopharyngitis	5.0% 4/80 • Number of events 5 • Day -28 to Week 54, a maximum of approximately 58 weeks	15.6% 12/77 • Number of events 13 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.3% 3/41 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Upper respiratory tract infection	0.00% 0/80 • Day -28 to Week 54, a maximum of approximately 58 weeks	9.1% 7/77 • Number of events 8 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.6% 1/39 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Urinary tract infection	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	9.1% 7/77 • Number of events 7 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/41 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Oral herpes	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	7.8% 6/77 • Number of events 7 • Day -28 to Week 54, a maximum of approximately 58 weeks	0.00% 0/39 • Day -28 to Week 54, a maximum of approximately 58 weeks	4.9% 2/41 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks
Infections and infestations Sinusitis	1.2% 1/80 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks	3.9% 3/77 • Number of events 3 • Day -28 to Week 54, a maximum of approximately 58 weeks	5.1% 2/39 • Number of events 2 • Day -28 to Week 54, a maximum of approximately 58 weeks	2.4% 1/41 • Number of events 1 • Day -28 to Week 54, a maximum of approximately 58 weeks

Additional Information

Apellis Clinical Trial Information Line

Apellis Pharmaceuticals, Inc

Phone: 1-833-284-6361

Email: clinicaltrials@apellis.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place